Albireo Enrolls First Patient in Phase 3 Clinical Trial of Odevixibat in Biliary Atresia

— BOLD is first-ever pivotal trial for biliary atresia —

— Trial expands odevixibat’s development program into second rare cholestatic liver disease —

BOSTON, July 14, 2020 (GLOBE NEWSWIRE) --  Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced the first patient has been enrolled in the BOLD (Biliary atresia and the use of Odevixibat in treating Liver Disease) global Phase 3 clinical trial of odevixibat, a highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), for the treatment of biliary atresia. There are no approved pharmacological treatments for biliary atresia which is the most common pediatric cholestatic liver disease and the leading cause of pediatric liver transplant across all diseases.  BOLD is the largest, prospective intervention trial ever conducted in biliary atresia, expanding the development of odevixibat to a second rare cholestatic liver disease indication.  Two clinical sites in the United States are currently active for patient enrollment in the second odevixibat Phase 3 pivotal trial.

Biliary atresia is a rare pediatric liver disease with symptoms typically developing about two to eight weeks after birth and no approved pharmacological therapies. Damaged or absent bile ducts outside the liver result in bile and bile acids being trapped inside the liver, quickly resulting in cirrhosis, and even liver failure. The disease impacts an estimated 15,000-20,000 people in the United States and European Union and is the leading cause of liver transplants among children. Odevixibat has received orphan drug designation for biliary atresia in the United States and European Union.

“The initiation of the BOLD trial is encouraging progress for biliary atresia patients, their families and clinicians, as it is the first-ever pivotal trial of a pharmacological therapy for this devastating rare disease,” said Saul Karpen, M.D., Ph.D., Pediatric Hepatologist at Children’s Healthcare of Atlanta and lead investigator of the BOLD trial. “There is an incredible unmet need for these patients with biliary atresia, since there are no effective therapies to stem the expected progression of disease. This lack of effective therapeutics leads to the current state of the field where approximately 70 percent of biliary atresia patients will need a liver transplant during childhood. I look forward to seeing how a targeted therapeutic, odevixibat, one that specifically addresses the accumulation of bile acids in the liver, may delay or prevent liver transplant for babies with this serious disease.”